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Swine Flu Information

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Swine Influenza (swine flu) is a respiratory disease of pigs caused by type A influenza that regularly cause outbreaks of influenza among pigs.

Swine flu viruses do not normally infect humans, however, human infections with swine flu do occur, and cases of human-to-human spread of swine flu viruses has been documented.

From December 2005 through February 2009, a total of 12 human infections with swine influenza were reported from 10 states in the United States. Since March 2009, a number of confirmed human cases of a new strain of swine influenza A (H1N1) virus infection in California, Texas, and Mexico have been identified.

Human Swine Influenza Investigation

April 25, 2009 19:30 EDT

Human cases of swine influenza A (H1N1) virus infection have been identified in the U.S. in San Diego County and Imperial County, California as well as in San Antonio, Texas. Internationally, human cases of swine influenza A (H1N1) virus infection have been identified in Mexico.

U.S. Human Cases of Swine Flu Infection
State # of laboratory confirmed cases
California 7 cases
Texas 2 cases
Kansas 2 cases
TOTAL COUNT 11 cases
International Human Cases of Swine Flu Infection
See: World Health Organization

Last Updated: As of April 25th, 2009 7:30 p.m. EDT

Investigations are ongoing to determine the source of the infection and whether additional people have been infected with similar swine influenza viruses.

CDC is working very closely with state and local officials in California, Texas, as well as with health officials in Mexico, Canada and the World Health Organization. On April 24th, CDC deployed 7 epidemiologists to San Diego County, California and Imperial County, California and 1 senior medical officer to Texas to provide guidance and technical support for the ongoing epidemiologic field investigations. CDC has also deployed to Mexico 1 medical officer and 1 senior expert who are part of a global team that is responding to the outbreak of respiratory illnesses in Mexico.

Influenza is thought to spread mainly person-to-person through coughing or sneezing of infected people. There are many things you can to do preventing getting and spreading influenza:

There are everyday actions people can take to stay healthy.

  • Cover your nose and mouth with a tissue when you cough or sneeze. Throw the tissue in the trash after you use it.
  • Wash your hands often with soap and water, especially after you cough or sneeze. Alcohol-based hands cleaners are also effective.
  • Avoid touching your eyes, nose or mouth. Germs spread that way.

Try to avoid close contact with sick people.

  • Influenza is thought to spread mainly person-to-person through coughing or sneezing of infected people.
  • If you get sick, CDC recommends that you stay home from work or school and limit contact with others to keep from infecting them.

Source: Centers for Disease Control (CDC)

Promising Procedure Injects Stem Cells Directly Into Patient’s Heart

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In a ground-breaking procedure, surgeons at The Methodist Hospital in Houston injected highly concentrated stem cells directly into a patient’s heart, providing an intense, direct hit on damaged heart tissue.

This technique may be more successful in regenerating healthy heart tissue than current methods that use a catheter to put standard stem cells through the bloodstream into the heart. The 58-year old patient is expected to be discharged this weekend.

“Some patients have such severe heart failure that their only current option is a heart transplant,” said Dr. Brian Bruckner, cardiac surgeon at the Methodist DeBakey Heart & Vascular Center in Houston. “We hope that stem cells will stimulate angiogenesis, the growth of new blood vessels, restore mechanical function in diseased heart tissue, and return patients to a much better quality of life without a transplant.”

In a novel process, the patient’s strongest and most robust stem and progenitor cells, derived from the patient’s own bone marrow, are amplified up to 1,000 times before they’re injected back into the patient’s heart. In the procedure, Dr. Bruckner made a small incision in the left side of the patient’s chest and administered approximately 25 injections of concentrated stem cells into the patient’s heart. All patients in the trial will be followed for 12 months after the injections.

There are currently 5.5 million people in the U.S. suffering from chronic heart failure. A subset of these patients has dilated cardiomyopathy (DCM), a chronic heart disease in which the patient’s heart can not pump effectively enough to deliver blood and oxygen to the vital organs in the body. Patients with DCM typically experience severe limitations to physical activity and shortness of breath.

“Without a new approach to treatment of these patients, they will continue to decline and less than 40 percent will survive five years,” said Bruckner, principal investigator for the trial. “We hope this trial will provide a completely new and viable treatment for them.”

Dr. Michael Reardon, chief of cardiac surgery at Methodist, and Dr. Matthias Loebe, transplant surgeon at Methodist, are co-investigators on the trial. Dr. Kevin Lisman is the patient’s referring cardiologist.

Source: Methodist Hospital, Houston, Texas

Trauma Victims’ Post-Operational Survival Chances Improved by Antioxidant Therapy

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Many trauma patients do not survive the post-operational stage due to multiple-organ failure, but a recent study has shown that post-operational survival can be improved by antioxidant therapy.

In a study presented at the 2008 Clinical Congress of the American College of Surgeons (ACS), Bryan A. Cotton, MD, FACS, stated that “implementation of high-dose antioxidant protocol (vitamins C, E, and selenium) resulted in a reduction of pulmonary complications, in general, as well as infectious complications, including central line and catheter-related infections.”

Dr. Cotton, assistant professor of surgery at Vanderbilt University Medical Center, Nashville, TN, observed that when an abdominal wound opens up, the result is not just an infection to be treated with antibiotics. Sometimes the wounds open up, requiring reconstruction with expensive agents. Dr. Cotton observed a remarkable decrease in abdominal wall complications—including abdominal compartment syndrome and surgical site infections.

“This is a high mortality, high morbidity, may-never-return-to-work-again problem in a young healthy patient,” he said. “Abdominal wall complications are enormous, yet we noted a reduction in some of these complications with implementation of antioxidants. Importantly, the biggest difference was in those patients who had a predicted mortality exceeding 50 percent.”

Dr. Cotton and his colleagues at Vanderbilt showed that this high-dose antioxidant protocol accounted for an amazing 28% reduction in mortality in acutely injured patients. Length-of-stay, hospital and ICU, was also reduced. Dr. Cotton explained how an acute injury imposes a huge strain on the body, which releases oxygen molecules called free radicals, that cause damage at the cellular level called oxidative stress. Antioxidants work as a team in mopping up some of the oxidative stress waste byproducts, reducing the stressors that cause harm. Depletion of antioxidants is one of the mechanisms that explains why we are vulnerable. Antioxidant therapy replenishes those troops to help keep us safe.

“Antioxidant therapy is so simple and that’s what throws people off,” Dr. Cotton said. A trial conducted by Avery B. Nathens, MD, MPH, showed that some inflammatory states and responses were remarkably improved in patients who had received antioxidants versus those who did not. “Based on these results, we were inspired to initiate a study with vitamins C and E. When we looked at the literature, however, there were some concurrent studies showing that selenium had an impact too, especially on sepsis and other infectious complications. So we combined all the existing research and did a cost analysis. When we learned it would cost only $11 a patient for a seven-day course of antioxidants, we decided to give it a try.”

This retrospective study followed a total of 4,279 patients admitted to the Vanderbilt University Medical Center trauma unit during the study period. High-dose antioxidant protocol was administered to all acutely injured patients (2,258 individuals) admitted to the center between October 1, 2005, and September 30, 2006. This treatment included 1,000 mg. vitamin C (ascorbic acid) -tocopherol acetate), each routinely given everyaand 1,000 IU vitamin E (DL- eight hours by mouth, if the patient could take it that way. In addition, 200 mcg. selenium was given once daily intravenously. Patients received these supplements upon arrival, and they were continued for seven days or until discharge, whichever happened first. Patients who were pregnant or had serum creatinine levels >2.5mg/dL did not receive antioxidants.

A comparison cohort was made up of all patients (2,021 individuals) admitted to the trauma center between October 1, 2004, and September 30, 2005—prior to implementation of the antioxidant protocol. While pneumonia and renal failure were similar between the groups, the incidence of abdominal compartment syndrome was significantly less (90 versus 31), as were catheter-related infections (75 versus 50) and surgical site infections (101 versus 44). Pulmonary failure—meaning the patient could not get off the ventilator—was less as well (721 versus 528).

Dr. Cotton is now prescribing high-dose antioxidants only to the most seriously ill patients in the ICU, as they seem to derive the greatest benefit. He and his colleagues will now focus on dose adjustments and length of administration to see if the doses and duration they are currently using are optimal. They have been approached by several groups that are interested in collaborating and investigating these agents as part of multiinstitutional trials and expanding their use to critically ill nontrauma patients.

“While we are all looking for that magic bullet to cure some of the horrible things that can happen after someone is injured or has an operation, we have something at our disposal,” Dr. Cotton said. “It might not be that magic bullet, but it is a very inexpensive and safe way to reduce complications and mortality in the sickest patients.”

Assisting Dr. Cotton with this study were Aviram Giladi, BS; Bryan R. Collier, DO, FACS; Lesly A. Dossett, MD; and Sloan B. Fleming, PharmD, all from Vanderbilt. He received no funding for this research.

Source: American College of Surgeons

Spotting The Difference Between Heartburn & Heart Attack Can Be A Lifesaver

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How many people regret over-indulging on Thanksgiving Day? Some can sleep off the tumult in their stomachs, others experience alarming symptoms. Is that pain in their chest heartburn or a heat attack? The symptoms are similar, but the health consequences differ dramatically.

“We see people in the Emergency Room who think they are only having severe heartburn or experiencing the flu when they are actually having a heart attack,” said Nick Zenarosa, M.D., emergency medicine physician on the medical staff at Baylor University Medical Center at Dallas.

If you think you are experiencing heartburn, Dr. Zenarosa recommends watching for the following symptoms which are not typical of heartburn and could indicate a heart attack:

  • Breaking into a cold sweat
  • Pain moving from the chest into the jaw, shoulder or arms
  • Increased pain when you exert yourself, rapid onset of fatigue
  • shortness of breath
  • turning pale
  • slow or no response of symptoms to antacids
  • nausea and possible vomiting

Keep in mind that the signs of a heart attack can be subtle, particularly in women. If you are experiencing any of these signs, coupled with chest pain and/or pain that radiates through your jaw or down your arm, be sure to go to an Emergency Room.

Time is of the essence when a person is having a heart attack. According to the National Heart, Lung and Blood Institute, the sooner clot-busting drugs and other artery-opening treatments are started, the more good they will do, and the greater the chances are for survival and a full recovery.

Source: Baylor Health Care System

Scientists Finding Anti-Obesity Drugs With Fewer Side Effects

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Fen-phen was once regarded as a miracle weight-loss drug, but when it induced heart valve lesions and mother life-threatening side effects, it was taken off the market. UT Southwestern Medical Center scientists are exploring Fen-phen’s behavior in the brain so that safer anti-obesity drugs with less side effects can be developed.

In a recent study, the researchers define a circuit in the brain that explains the ways fenfluramine, a component of Fen-phen, suppresses appetite.

“Our findings provide evidence that the neural circuit we’ve proposed is sufficient for the neurotransmitter serotonin to regulate food intake and body weight, ” said Dr. Joel Elmquist, professor of internal medicine and pharmacology at UT Southwestern and senior author of the study. “Fen-phen works directly on this pathway. Unfortunately, that drug also adversely affects peripheral tissue such as the heart.”

Mice were engineered for the current study, in which the expression of a serotonin receptor called 5-hydroxytryptamine 2C was blocked throughout the entire body. Previously, this produced obese mice resistant to the anorexic actions of fenfluramine. When activated by serotonin, however, this receptor is also known to suppress appetite. Using this mouse model, the authors engineered another set of mice in which the same serotonin receptor was blocked everywhere in the body except within a group of brain cells called pro-opiomelanocortin, or POMC, neurons. The POMC neurons, which are found in the hypothalamus, are also known to play an important role in suppressing appetite and inducing weight loss.

The scientists noted that the animals with no serotonin 2c receptors expectedly developed obesity as well as other metabolism disorders such as increased food intake, hyperactivity and leptin insensitivity. They also were prone to spontaneous seizures, said Dr. Elmquist. The mice in which the serotonin receptor was fre-expressed and functioning only in the POMC neurons stayed slim and responded to fenfluramine.

“The POMC-specific reactivation of the receptor only in POMC neurons normalizes the abnormal metabolism in these mice,” Dr. Elmquist said. “The animals don’t eat excessively. Their hyperactivity is also gone.”

Previous work from the UT Southwestern group led to the hypothesis that Fen-phen worked by activating the serotonin 2c receptor in the POMC neurons in the hypothalamus. The current work provides genetic proof supporting this model.

“Conventional wisdom is that fenfluramine increases serotonin release that then activates serotonin receptors in the brain to regulate food intake and body weight, but unfortunately, this drug also causes lesions in heart valves,” he said. “If you could develop a drug that would travel to both the brain and the peripheral tissues, and then give a blocker to protect the heart, it’s possible that you could prevent the harmful side effects and still aid weight loss. Admittedly, that’s a bit farfetched, but this mouse model could be used to test that theory.”

The team’s next step is to determine whether they’ve identified the sole circuit required to suppress appetite and induce weight loss.

Source: Neuron,

Chronic Kidney Disease Up 30% Over Past Decade

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A 30 percent increase in chronic kidney disease over the past decade has prompted the U.S. Renal Data System (USRDS) to issue for the first time a separate report documenting the magnitude of the disease, which affects an estimated 27 million Americans and accounts for more than 24 percent of Medicare costs.

“NIDDK’s annual analysis and publication of data on kidney disease in the United States is essential in quantifying public health trends, guiding funding priorities, and designing targeted kidney research programs,” said NIH Director Elias A. Zerhouni, M.D. “The major focus on chronic kidney disease in this year’s report acknowledges that this disorder is a growing public health issue deserving of wider public awareness and intensified scientific investigation.”

Using data from multiple sources, the USRDS has created a new handbook of information that can be used by researchers, government officials, health program planners, and others to develop research goals, assess public health needs, set program priorities, and inform policymakers and the public. USRDS research depends on collaborations with other agencies of the U.S. Department of Health and Human Services, especially the Centers for Medicare and Medicaid Services, the Health Resources and Services Administration, and the Centers for Disease Control and Prevention. Patient registries for other countries also contribute data for analyses.

Volume One of the report defines the disease burden of chronic kidney disease and examines cardiovascular and other related health problems, rates of adverse health events, preventive care, prescription medication therapies, delivery of care in the transition to end-stage renal disease, and the cost to Medicare and employer group health plans.

One of the major findings central to public health is that those with chronic kidney disease are more likely to die from cardiovascular disease than to reach end-stage kidney disease. However, cardiovascular risk factors can be detected and treated. This suggests that those transitioning from chronic to end-stage kidney disease merit more attention. Expenditures during the transition from chronic to end-stage kidney disease are considerable, ranging from $14,500 for Medicare patients to $29,000 for those covered by employer group health plans in the month of dialysis initiation.

“These latest data on kidney disease underscore the importance of the research we fund,” said NIDDK Director Griffin P. Rodgers, M.D. “With rising rates of chronic and end-stage kidney disease, we need to stimulate research that will help us discover new, effective therapies for these devastating disorders.”

Volume Two reports that the number of people with end-stage kidney disease is increasing in size and cost. The incidence of chronic kidney disease in 2006 was more than 100,000, or 360 per one million people, an increase of 3.4 percent over the 2005 incidence rate. There were more than half a million patients with end-stage kidney disease in 2006. Of these, 70 percent were on dialysis. An important step before a patient begins dialysis is the preparation of a vascular access, which is the site on the patient’s body where blood is removed and returned during dialysis.

The three types of vascular access for dialysis are arteriovenous (AV) fistula, an AV graft, and a venous catheter. Both the fistula and the graft involve connecting an artery to a vein, usually beneath the skin in a patient’s arm.

The fistula is considered the best long-term vascular access for dialysis. The catheter is a tube inserted into a vein in the patient’s neck, chest, or leg near the groin. It is usually only used as a temporary access until a permanent fistula or graft can be developed. This volume reports that more than 80 percent of new dialysis patients started with a catheter, more than 50 percent of current dialysis patients had a fistula, and 30 percent had a graft.

Volume Two also reports that Medicare paid about $70,000 per dialysis patient. Patients with end-stage kidney disease accounted for a little more than 1 percent of the Medicare population and more than 7 percent of Medicare costs. Total cost for end-stage kidney disease was $33.6 billion. This number includes Medicare spending and all expenditures by other payers, such as employer group health plans.

In addition, more than 18,000 kidney transplants were performed in 2006, an increase of 3.5 percent over 2005. Use of deceased donor kidneys increased between 2003 and 2006 at a rate of about 6 percent to 7 percent. Use of living donors fell 3 percent during that period, but the use of living unrelated donors continues to increase relative to the total number of living donations, and now accounts for 45 percent of all living donor transplantations.

NIDDK conducts and supports research in diabetes and other endocrine and metabolic diseases; digestive diseases, nutrition, and obesity; and kidney, urologic, and hematologic diseases. Spanning the full spectrum of medicine and afflicting people of all ages and ethnic groups, these diseases encompass some of the most common, severe, and disabling conditions affecting Americans.

The USRDS is funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health (NIH). The USRDS 2008 Annual Data Report: Atlas of Chronic Kidney Disease and End-Stage Renal Disease, is online at www.usrds.org.

The National Institutes of Health (NIH) — The Nation’s Medical Research Agency — includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

Source: National Institutes of Health (NIH)

Women More Vulnerable to Alcohol’s Long-term Effects Than Men

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Women are more vulnerable to alcohol’s longterm effects than men, according to the Harvard Heart Letter. The difference is in women’s ability to break down alcohol, which is slower than men’s.

The result is that a woman drinking the same amount as a man will have a higher blood level of alcohol, and for a longer time. Her tissues are exposed to more alcohol per drink than a man’s, and a Japanese study indicates that too much alcohol is bad for a woman’s heart and arteries, as well as being a danger to breast tissue.

Current thinking, according to the Harvard Heart Letter, suggests that “healthy drinkng” is no more than two drinks daily for men and one drink daily for women, a recommendation for the average person. The effects of alcohol intake will depend on your genes, diet and medications taken. Since alcohol prevents the absorption of folic acid, drinkers need to take extra folic acid, which can be accomplished by taking a daily multivitamin/multimineral supplement.

Source: Harvard Heart Letter

Americans Consume Twice Daily Recommendations of Salt

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Most Americans consume more than double the amount of their daily recommended level of sodium. A new study by the Centers for Disease Control and Prevention shows that more than 2 out of 3 adults are in population groups that should consume no more than 1,500 milligrams (mg) per day of sodium. During 2005-2006 the estimated average intake of sodium for persons in the United States age 2 years and older was 3,436 mg per day.

A diet high in sodium increases the risk of having higher blood pressure, a major cause for heart disease and stroke. These diseases are the first and third leading causes of death in the United States.

“It’s important for people to eat less salt. People who adopt a heart healthy eating pattern that includes a diet low in sodium and rich in potassium and calcium can improve their blood pressure,” said Darwin R. Labarthe, M.D., Ph.D., director of the CDC’s Division for Heart Disease and Stroke Prevention. “Reducing sodium intake can prevent or delay increases in blood pressure for everyone.’’

“People need to know their recommended daily sodium limit and take action to reduce sodium intake,” Labarthe said. Most of the sodium we eat comes from packaged, processed and restaurant foods. CDC along with other HHS agencies, including the Food and Drug Administration, will be working with major food manufacturers and chain restaurants to reduce sodium levels in the food supply.

The study in CDC’s Morbidity and Mortality Weekly Report used data from the National Health and Nutrition Examination Survey, a survey designed to assess the health and nutritional status of adults and children in the United States.

This study is the first to use national data to show that 69.2 percent of the adult population belongs to a specific group that should aim to consume no more than 1,500 mg of sodium per day. This group includes persons with high blood pressure, blacks, or middle-aged and older adults (more than 40 years old). The 2005 Dietary Guidelines for Americans recommend that adults in general should consume less than 2,300 mg (approximately one teaspoon of salt) of sodium per day.

The dietary guidelines, by the U.S. Department of Health and Human Services and the U.S. Department of Agriculture, provide advice for people 2 years and older about how good dietary habits can promote health and reduce risk for major chronic diseases.

Nationwide, 16 million men and women have heart disease and 5.8 million are estimated to have had a stroke. People who reduce their sodium consumption benefit from improved blood pressure and reduce their risk for developing other serious health problems. Choosing foods like fresh fruits and vegetables, when eating out, asking that foods be prepared without added salt, and reading the nutrition label of foods before purchasing can improve health for all adults.

Source: CDC, March 26, 2009

Bacterial Infections

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Sometimes, Death Is Good….. For A Vicious Unicellular Microorganism

There are a variety of different types of foreign bacterial infections one can get from many different sources, yet some are more common than others. If they are not beneficial for your physiology, they all should die in order to restore your health.
Bacteria are a simple life form, yet are incredibly productive and efficient. As with other life forms, it exists to reproduce, and does so about every hour, and evolves and adapts to its environment as needed. To do this, it fully utilizes all available resources and energy to develop the protein that is essential for its survival, and bacteria have the ability to adapt as needed to assure this happens.
It needs exactly 7 genes to produce the essential ribosomes for this to occur. Any more or less genes than 7, the bacteria is not maximizing its efficiency to survive and reproduce. Amazing.
Strept infections are caused by what are called gram positive bacteria, and are unique that these bacteria grow in pairs. Staph bacterial invasions are gram positive as well, yet it is the MRSA, Methicillin Resistant Staff Aureous microbes of this type often are very difficult to treat normally when a patient suffers from their damage from being invaded by these bacteria. Another difficult situation is when a patient is infected by VRE, Vancomycin Resistant Enterococci, as well.
These MRSA and VRE pathogenic or disease causing bacteria are the ones that are the most clinically concerning for the health care provider.
Group A strep infections can cause diseases such as strep throat and pneumonia. Since there are several types of bacteria, a diagnostic test called a culture and sensitivity is usually performed to assure the correct antibiotic is selected for treatment, as the bacteria are identified with this method.
Typically, fluid from the area suspected of being infected is obtained from the patient suspected to have an infection and smeared on what is called a petrie dish. And then these dishes are incubated for 2 to 3 days. Gram positive bacteria stain during this process a dark violet or blue. Gram negative bacteria would be pink in color, and are capable of harm as well to a human being.
When the culture is complete, technology offers recommendations on the appropriate class or brand of antibiotic for this bacteria present in another person- presuming the bacteria will not be resistant to the antibiotic recommended, as this happens on occasion.
Usually, classes of antibiotics that are used to treat gram positive strep infections that are not VRE or MRSA are cephalosporins, macrolides, or general penicillins. If the microbe that is causing the infection is resistant to the antibiotic from such classes that are administered to the infected patient, particularly with methicillin and vancomycin, which is the case with VRE and MRSA bacteria, then there are other more aggressive antibiotics that will be chosen for this patient.
Such brands and types of antibiotics for MRSA and VRE bacteria include Zyvox, which has both IV and oral dosage options. There are also other antibiotics, such as Cubicin. However these antibiotics for antibiotic resistant bacteria are given usually due to infections that have progressed to a more serious nature within a patient infected in such a way.
Progressive medical conditions include sepsis, or blood infection, osteomyelitis, or bone infection, or Pneumonia, which is a serious lung infection. A hospital stay is normally required with such patients, as the last antibiotics mentioned for MRSA and VRE bacterial infections are given by IV administration initially for several days, if not several weeks.
There are numerous classes and types of antibiotics available, yet bacterial resistance to most of these antibiotics constantly remains serious concern for the health care provider, and the infected patient, with MRSA at the top of the list of concerns for the health care providers.
Dan Abshear
http://www.cdc.gov/ncidod/dhqp/ar_mrsa_spotlight_2006.html

Should Patients Have Access to Investigational Unapproved Drugs?

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As a cancer physician, I often cared for patients who found themselves in very desperate medical situations. When all available approved treatment alternatives have been exhausted, many patients are willing to try investigational or restricted therapies- -but then learn they are not eligible for the protocols that govern their use. What are such desperate patients to do?

My take is that once approved treatment regimens have been exhausted, desperately ill patients should have appropriate access to any reasonable treatment that has the potential to be of benefit and yet doesn’t present unjustifiable risks. The best way to provide these drugs is through clinical trials, where the rigor and discipline of the trial enables patient response, side effects, and outcomes to be carefully measured to determine safety and effectiveness in a systematic way. When this is not possible, there can be an alternative pathway to receiving the investigational therapy.

The FDA has called this pathway “single patient access,” often referred to by the public as ” compassionate use.” We have a long history of helping patients gain access to potential treatments this way. For example, just in FDA’s oncology division, we review and approve hundreds of requests each year for single patient access to investigational drugs.

More recently, we have encountered circumstances in which a drug is approved and is commercially available, but can only be used under very strict rules – these are called an approved Risk Evaluation and Mitigation Strategy (REMS). But we have adapted “single patient expanded access” to this category of drugs as well in an effort to do everything appropriate to serve these unfortunate patients.

So, how can patients gain access to an investigational drug or a restricted drug through these mechanisms? To permit such treatment use of an investigational drug, the company must first agree to make the drug available. Then, the FDA, in conjunction with the patient’s treating physician, must determine, among other things that the potential benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or the condition to be treated; and that providing the investigational drug for the treatment use will not interfere with the initiation, conduct, or completion of clinical investigations that are required to support marketing approval of the investigational drug for the greatest number of patients who can benefit. A similar path is taken when considering use of restricted commercially available drugs.

Drugs under investigation, in either a clinical trial or an expanded access program, don’t offer a guarantee of success, but they do offer an option. Information about clinical trials of investigational drugs and expanded access options, including contacts and locations, is available through the ClinicalTrials.gov web site. At the FDA, there is an Office of Special Health Issues which you can call and where they have trained personnel to listen to your story and assist you with the necessary information. The number to call is 301-827-4460.

At the end of the day, benefitting patients is the goal of the FDA. And how it is achieve requires a structured and disciplined process to facilitate getting the right drug or medical product to the right patient in the right way – to get the best possible outcome.

Andrew C. von Eschenbach, M.D.
Commissioner of Food and Drugs (FDA)